Haydar Frangoul, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Lyndsay Molinari, Donna Wall, Robert I Liem, Paul Telfer, Ami J Shah, Marina Cavazzana, Selim Corbacioglu, Damiano Rondelli, Roland Meisel, Laurence Dedeken, Stephan Lobitz, Mariane de Montalembert, Martin H Steinberg, Mark C Walters, Michael J Eckrich, Suzan Imren, Laura Bower, Christopher Simard, Weiyu Zhou, Fengjuan Xuan, Phuong Khanh Morrow, William E Hobbs, Stephan A Grupp
BACKGROUND: Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) at the erythroid-specific enhancer region of BCL11A . METHODS: We conducted a phase 3, single-group, open-label study of exa-cel in patients 12 to 35 years of age with sickle cell disease who had had at least two severe vaso-occlusive crises in each of the 2 years before screening...
April 24, 2024: New England Journal of Medicine